Patent classifications
A61K31/7088
METHODS FOR THE TREATMENT OF TRINUCLEOTIDE REPEAT EXPANSION DISORDERS ASSOCIATED WITH MLH3 ACTIVITY
The present disclosure features useful compositions and methods to treat repeat expansion disorders, e.g., in a subject in need thereof. In some aspects, the compositions and methods described herein are useful in the treatment of disorders associated with MLH3 activity.
DEVELOPMENT OF DUAL-gRNA APPROACH WITH UNDETECTABLE OFF-TARGET EFFECT TO CORRECT C9ORF72 REPEAT EXPANSION AND C9ORF72 PATHOLOGY
A group of gRNAs is provided, and the group of gRNAs comprises a first gRNA molecule; and a second gRNA molecule capable of defining a region in a genome sequence with the first gRNA molecule, wherein the region in the genome comprises a target sequence in need of removal.
DEVELOPMENT OF DUAL-gRNA APPROACH WITH UNDETECTABLE OFF-TARGET EFFECT TO CORRECT C9ORF72 REPEAT EXPANSION AND C9ORF72 PATHOLOGY
A group of gRNAs is provided, and the group of gRNAs comprises a first gRNA molecule; and a second gRNA molecule capable of defining a region in a genome sequence with the first gRNA molecule, wherein the region in the genome comprises a target sequence in need of removal.
MUTANT KLF PROTEIN, AND METHOD FOR PRODUCING INDUCED PLURIPOTENT STEM CELLS
There is provided a mutant KLF protein that can induce reprogramming of a somatic cell at a higher efficiency than a KLF protein having a natural amino acid sequence. There is also provided a method for efficiently producing an iPS cell by using the mutant KLF protein. There is provided a mutant KLF protein having an amino acid substitution, or a peptide fragment thereof containing the amino acid substitution.
VACCINATION AGAINST ANTIGENS INDUCED IN PATHOGEN-INFECTED CELLS
The present invention relates, in part, to methods of generating immune responses in subjects to treat an infectious disease.
VACCINATION AGAINST ANTIGENS INDUCED IN PATHOGEN-INFECTED CELLS
The present invention relates, in part, to methods of generating immune responses in subjects to treat an infectious disease.
Treatment Of Psoriasis With Interferon Induced Helicase C Domain 1 (IFIH1) Inhibitors
The present disclosure provides methods of treating subjects having psoriasis, and methods of identifying subjects having an increased risk of developing psoriasis.
Anti-aging agent for skin and anti-aging-related gene expression regulator
The present invention has an object to provide a skin anti-aging agent, an agent for regulating expression of anti-aging related gene, and a cosmetic product comprising the anti-aging agent or the agent for regulating expression of anti-aging related gene which are very safe and can be used reliably for a long term. The skin anti-aging agent, the agent for regulating expression of anti-aging related gene, and the cosmetic product comprising the anti-aging agent or the agent for regulating expression of anti-aging related gene of the present invention comprise a special low molecular DNA and a soybean extract, preferably a soybean sprout extract, as active ingredients. These active ingredients have activating actions on the function of the dermal fibroblast and are hence expected to prevent or improve the skin aging such as the reduction of skin firmness and elasticity, wrinkles and sagging.
Anti-aging agent for skin and anti-aging-related gene expression regulator
The present invention has an object to provide a skin anti-aging agent, an agent for regulating expression of anti-aging related gene, and a cosmetic product comprising the anti-aging agent or the agent for regulating expression of anti-aging related gene which are very safe and can be used reliably for a long term. The skin anti-aging agent, the agent for regulating expression of anti-aging related gene, and the cosmetic product comprising the anti-aging agent or the agent for regulating expression of anti-aging related gene of the present invention comprise a special low molecular DNA and a soybean extract, preferably a soybean sprout extract, as active ingredients. These active ingredients have activating actions on the function of the dermal fibroblast and are hence expected to prevent or improve the skin aging such as the reduction of skin firmness and elasticity, wrinkles and sagging.
SMAC/Diablo inhibitors useful for treating cancer
The present invention relates to compositions and methods for treating cancer, particularly to agents that inhibit the expression and/or activity of the protein second mitochondria-derived activator of caspase/direct inhibitor of apoptosis-binding protein with low pI (SMAC/Diablo). The inhibiting agents include RNA interference molecules silencing the expression of SMAC/Diablo and peptides modulating its interactions within the cell nucleus and mitochondria. The methods and agents of the present invention are useful in treating cancers associated with overexpression of SMAC/Diablo.